In the phase 3 clinical trial CLIMB SCD-121, which evaluated exagamglogene autotemcel (exa-cel) in severe sickle cell disease (SCD), exa-cel produced marked quality of life (QoL) improvements in patients who had at least 16 months of follow-up. These benefits, which authors describe in a trial analysis published in Blood Advances, are in addition to 97% of the enrolled patients having achieved vaso-occlusive crisis (VOC) elimination via the cell therapy.
“These results confirm the broad clinical benefits of exa-cel, including associated improvements in quality of life and reductions in pain experience, in patients with severe SCD,” wrote first author for the analysis, Akshay Sharma, MBBS, MSc, of St Jude Children’s Research Hospital, Memphis, Tennessee, and colleagues.
The investigators evaluated patient-reported outcome (PRO) measures for 30 adult patients at 6 months after receipt of exa-cel infusion. By that point, these patients had achieved significant improvement in their EuroQol Quality of Life Scale-5 dimensions-5 levels of severity (EQ-5D-5L) health utility index scores for the United States and United Kingdom, as well as in their EuroQol Visual Analog Scale (VAS) scores; benefits which lasted through the 36th month after infusion. Comparable improvement was also seen in the patients’ bone marrow transplant subscale (BMTS) and Functional Assessment of Cancer Therapy-General (FACT-G) total scores.